Community healthcare appointments as an alternative to emergency department assessment: an exploration of family acceptability and preferences.

PURPOSE: Pediatric emergency departments are overcrowded, in part due to many non-emergent visits. We aimed to assess the proportion of parents interested in leaving the pediatric emergency department (ED) prior to physician assessment if they could be offered a scheduled community healthcare appointment. We explored differences in care children received in the ED stratified by interest in a community healthcare appointment and parents' reasons when they were not interested. METHODS: We conducted a 14-item survey within the pediatric ED at a Canadian tertiary care teaching hospital to assess parents' interest if a program offered community healthcare appointments and we determined preferred appointment characteristics. All parents presenting with children triaged as CTAS 2-5 who met eligibility criteria were approached by a research assistant prior to physician assessment. Surveys were paired with the medical chart outlining the care received. Descriptive statistics and a regression model were used to describe characteristics of families and care received among those who were and were not interested in a community healthcare appointment. RESULTS: In total, 403 surveys were completed. Overall, 236 participants (58.6%; 95% CI 53.8-63.4) were interested in a community healthcare appointment. In general, parents who were interested in a community healthcare appointment were younger and presented with younger children compared to those who were not interested. Among those interested, there was a preference to have the appointment with a pediatrician or family physician, timely access to an appointment, and appointments scheduled outside of regular business hours. CONCLUSION: Our study provides evidence that there is interest in an alternative care access model positioned to reduce pediatric ED congestion. We found that parents would be interested in leaving the pediatric ED in favor of a community healthcare appointment, provided it was with a physician and available in a timely manner.

RéSUMé: OBJECTIF: Les services d'urgences pédiatriques sont surchargés, en partie à cause des nombreuses visites non urgentes. Nous avons cherché à évaluer la proportion de parents désireux de quitter le service des urgences pédiatriques avant l'évaluation du médecin si on leur proposait un rendez-vous dans un centre de soins de santé communautaire. Nous avons étudié les différences dans les soins reçus par les enfants aux urgences en fonction de leur intérêt pour un rendez-vous dans un centre de soins de santé communautaire et des raisons invoquées par les parents lorsqu'ils n'étaient pas intéressés. MéTHODES: Nous avons mené une enquête de 14 points au sein du service des urgences pédiatriques d'un hôpital universitaire canadien de soins tertiaires afin d'évaluer l'intérêt des parents pour un programme offrant des rendez-vous de soins de santé communautaires et nous avons déterminé les caractéristiques des rendez-vous préférés. Tous les parents se présentant avec des enfants triés selon l'ETG 2­5 et répondant aux critères d'éligibilité ont été approchés par un assistant de recherche avant l'évaluation par le médecin. Les questionnaires ont été associés au dossier médical décrivant les soins reçus. Des statistiques descriptives et un modèle de régression ont été utilisés pour décrire les caractéristiques des familles et les soins reçus parmi ceux qui étaient et n'étaient pas intéressés par un rendez-vous en soins de santé communautaire. RéSULTATS: Au total, 403 enquêtes ont été réalisées. Dans l'ensemble, 236 participants (58,6%; IC à 95% 53,8­63,4) étaient intéressés par un rendez-vous en soins de santé communautaires. En général, les parents intéressés par un rendez-vous dans les soins de santé communautaires étaient plus jeunes et se présentaient avec des enfants plus jeunes que ceux qui n'étaient pas intéressés. Parmi les personnes intéressées, on note une préférence pour un rendez-vous avec un pédiatre ou un médecin de famille, un accès rapide à un rendez-vous et des rendez-vous fixés en dehors des heures normales de bureau. CONCLUSIONS: Notre étude montre qu'il existe un intérêt pour un modèle d'accès aux soins alternatif destiné à réduire l'engorgement des urgences pédiatriques. Nous avons constaté que les parents seraient intéressés à quitter le service d'urgence pédiatrique en faveur d'un rendez-vous de soins de santé communautaires pourvu qu'il soit avec un médecin et disponible en temps opportun.

Assessing the reliability of pediatric emergency medicine billing code assignment for future consideration as a proxy workload measure.

OBJECTIVES: Prediction of pediatric emergency department (PED) workload can allow for optimized allocation of resources to improve patient care and reduce physician burnout. A measure of PED workload is thus required, but to date no variable has been consistently used or could be validated against for this purpose. Billing codes, a variable assigned by physicians to reflect the complexity of medical decision making, have the potential to be a proxy measure of PED workload but must be assessed for reliability. In this study, we investigated how reliably billing codes are assigned by PED physicians, and factors that affect the inter-rater reliability of billing code assignment. METHODS: A retrospective cross-sectional study was completed to determine the reliability of billing code assigned by physicians (n = 150) at a quaternary-level PED between January 2018 and December 2018. Clinical visit information was extracted from health records and presented to a billing auditor, who independently assigned a billing code-considered as the criterion standard. Inter-rater reliability was calculated to assess agreement between the physician-assigned versus billing auditor-assigned billing codes. Unadjusted and adjusted logistic regression models were used to assess the association between covariables of interest and inter-rater reliability. RESULTS: Overall, we found substantial inter-rater reliability (AC2 0.72 [95% CI 0.64-0.8]) between the billing codes assigned by physicians compared to those assigned by the billing auditor. Adjusted logistic regression models controlling for Pediatric Canadian Triage and Acuity scores, disposition, and time of day suggest that clinical trainee involvement is significantly associated with increased inter-rater reliability. CONCLUSIONS: Our work identified that there is substantial agreement between PED physician and a billing auditor assigned billing codes, and thus are reliably assigned by PED physicians. This is a crucial step in validating billing codes as a potential proxy measure of pediatric emergency physician workload.

Psychometric properties and informant agreement of the WHODAS 2.0 in youth with mental disorder.

Objective: This study examined psychometric properties, parent-youth agreement, and factors associated with agreement on the 12-item and 36-item versions of the World Health Organization Disability Assessment Schedule (WHODAS) 2.0. Methods: Data come from a clinical sample of 56 youth, aged 14-17 years, receiving mental health care at a pediatric hospital. Correlations between the WHODAS 2.0, KIDSCREEN-27, and demographic variables were used to assess validity. Internal consistency was measured using ordinal alpha. The Bland-Altman method and intraclass correlation coefficients (ICC) were used to assess parent-youth agreement. Logistic regression examined factors associated with disagreement > 0.5 standard deviation. Results: For both parent and youth, correlations were low to moderate in exploring convergent (τ= -0.42 to 0.01) and divergent validity (τ/r = -0.12 to 0.32). Internal consistency was adequate (α > 0.7). Parent WHODAS 2.0 scores were significantly lower than youth scores and Bland-Altman plots revealed poor parent-youth agreement (ICC = -0.04 to 0.33). Lower household income was associated with lower odds of disagreement on the 35-item WHODAS 2.0 (OR= 0.28, 95% CI= 0.08-0.99), and older youth age was associated with lower odds of disagreement on the 12-item WHODAS 2.0 (OR= 0.40, 95% CI= 0.19-0.84). Conclusion: The psychometric properties of both WHODAS 2.0 versions were similar, so the abbreviated version may be sufficient to measure functional impairment in a clinical context. Additional research is needed to better understand the factors that influence discrepancies between informants and the implications for care. However, reports from both youth and parents appear valuable in understanding functional impairment.

Objectif: La présente étude a examiné les propriétés psychométriques, l'entente parent-jeune, et les facteurs associés à l'entente sur les versions en 12 items et en 36 items de la World Health Organization Disability Assessment Schedule (WHODAS) 2.0 (calendrier d'évaluation du handicap de l'OMS). Méthodes: Les données proviennent d'un échantillon clinique de 56 jeunes, de 14 à 17 ans, qui reçoivent des soins de santé mentale dans un hôpital pédiatrique. Les corrélations entre le WHODAS 2.0, KIDSCREEN-27, et les variables démographiques ont servi à évaluer la validité. La cohérence interne était mesurée à l'aide d'alpha ordinal. La méthode Bland-Altman et les coefficients de corrélation interclasse (CIC) ont servi à évaluer l'entente parent-jeune. La régression logistique a examiné les facteurs associés à la mésentente de > 0,5 déviation standard. Résultats: Pour les parents et les jeunes, les corrélations étaient de faibles à modérées en explorant la validité convergente (τ= −0,42 à 0,01) et divergente (τ/r = −0,12 à 0,32). La cohérence interne était adéquate (α > 0,7). Les scores des parents au WHODAS 2.0 étaient significativement plus faibles que les scores des jeunes et les tracés Bland-Altman révélaient une mauvaise entente parent-jeune (CIC = −0,04 à 0,33). Le revenu du ménage plus faible était associé avec des probabilités plus faibles de mésentente au WHODAS 2.0 de 35 items (RC = 0,28, IC à 95 % = 0,08 à 0,99), et l'âge avancé du jeune était associé à des probabilités plus faibles de mésentente au WHODAS 2.0 de 12 items (RC = 0,40, IC à 95 % = 0,19 à 0,84). Conclusion: Les propriétés psychométriques des deux versions du WHODAS 2.0 étaient semblables, donc la version abrégée peut suffire à mesurer la déficience fonctionnelle dans un contexte clinique. Il faut une recherche additionnelle pour mieux comprendre les facteurs qui influencent les divergences entre informateurs et les implications dans les soins. Cependant, les rapports tant des jeunes que des parents semblent valables pour comprendre la déficience fonctionnelle.

Do female and male patients derive similar benefits from approved systemic oncology therapies? A systematic review and meta-analysis.

PURPOSE: The National Institutes of Health's policy for the inclusion of females in clinical research was a pivotal step towards the consideration of sex as a biological variable, which is of particular importance in oncology, given differential incidence and outcomes of cancer between the sexes, and known pharmacodynamic, pharmacokinetic, and immunological differences. Therefore, we aim to investigate if such biological sex-based differences translate to clinically meaningful outcome differences from recently approved systemic oncology therapies. METHODS: A systematic review of randomized control trials (RCTs) cited in Food and Drug Administration, European Medicines Agency, and Health Canada approvals was conducted. Chemotherapy, targeted agents, and immunotherapy RCTs reporting sex-based sub-group analyses for overall/progression-free survival (OS/PFS) were considered. Hazard ratios (HRs) and 95% confidence intervals (CIs) were utilized. Sensitivity analyses for survival endpoints, drug type, and cancer site were conducted. RESULTS: Ninety-nine RCTs were included, representing 62,384 patients (23,574 (38%) female). Pooled OS HRs [95% CIs] were 0.77 [0.72-0.81] and 0.76 [0.72-0.79] for females and males, respectively (P = 0.73), and 0.51 [0.47-0.56] and 0.57 [0.53-0.61] (P = 0.08) for PFS. Sensitivity analyses yielded similar results. No RCTs reported sex-based toxicity or quality-of-life (QOL) data. CONCLUSION: Female and male patients appear to derive comparable benefits from recently approved systemic oncology therapies. Future RCTs are encouraged to report sex-based toxicity and QOL data.

Implementing Technology Literacy Programs in Retirement Homes and Residential Care Facilities: Conceptual Framework.

BACKGROUND: The COVID-19 pandemic caused widespread societal disruption, with governmental stay-at-home orders resulting in people connecting more via technology rather than in person. This shift had major impacts on older adult residents staying in retirement homes and residential care facilities, where they may lack the technology literacy needed to stay connected. The enTECH Computer Club from the University of Waterloo in Ontario, Canada created a knowledge translation toolkit to support organizations interested in starting technology literacy programs (TLPs) by providing guidance and practical tips. OBJECTIVE: This paper aimed to present a framework for implementing TLPs in retirement homes and residential care facilities through expanding on the knowledge translation toolkit and the framework for person-centered care. METHODS: Major concepts relating to the creation of a TLP in retirement homes and residential care facilities were extracted from the enTECH knowledge translation toolkit. The domains from the framework for person-centered care were modified to fit a TLP context. The concepts identified from the toolkit were sorted into the three framework categories: "structure," "process," and "outcome." Information from the knowledge translation toolkit were extracted into the three categories and synthesized to form foundational principles and potential actions. RESULTS: All 13 domains from the framework for person-centered care were redefined to shift the focus on TLP implementation, with 7 domains under "structure," 4 domains under "process," and 2 domains under "outcome." Domains in the "structure" category focus on developing an organizational infrastructure to deliver a successful TLP; 10 foundational principles and 25 potential actions were identified for this category. Domains in the "process" category focus on outlining procedures taken by stakeholders involved to ensure a smooth transition from conceptualization into action; 12 foundational principles and 9 potential actions were identified for this category. Domains in the "outcome" category focus on evaluating the TLP to consider making any improvements to better serve the needs of older adults and staff; 6 foundational principles and 6 potential actions were identified for this category. CONCLUSIONS: Several domains and their foundational principles and potential actions from the TLP framework were found to be consistent with existing literatures that encourage taking active steps to increase technology literacy in older adults. Although there may be some limitations to the components of the framework with the current state of the pandemic, starting TLPs in the community can yield positive outcomes that will be beneficial to both older adult participants and the organization in the long term.

A Standpoint Approach to Return-to-Work Coordination: Understanding Union Roles.

Purpose This study examined how the participation of union representatives impacted return-to-work (RTW) processes, and explored key activities undertaken by union representatives involved in return-to-work coordination. Methods Forty-seven RTW coordinators (RTWCs) participated in in-depth, semi-structured interviews in 2018 as part of a cross-Canadian study investigating their strategies for managing challenges in the RTW process. The study included RTWCs from a variety of organisation types, including unionized organizations. Audio-recordings were transcribed, coded, and analysed using constant case comparison and deviant case analysis leading to the development of findings themes. Results Our findings highlight the role of union representatives in RTW processes and how their activities are seen by other parties involved with work accommodation. First, we describe Union RTWC's administrative functions and the extent of their involvement in RTW accommodation negotiations. Second, we examine how Union and Non-union RTWCs framed the same RTW processes differently, according to their own accountabilities. Finally, we identify the positive ways that union participation figured into the RTW process, including playing a role in identifying viable modified work and serving as a trusted party to help reluctant workers engage with RTW plans. Conclusions We introduce a standpoint perspective to shed light on how Union and Non-union RTWCs approached accommodation issues and consider acknowledgement of power relations as a starting point for managing divergent interests.

Parent-Child Agreement on the Mini International Neuropsychiatric Interview for Children and Adolescents (MINI-KID).

OBJECTIVE: Multiple informants are often used in the assessment of child psychopathology; however, parent-child agreement is low in child psychiatry. The objective of this exploratory study was to assess informant agreement on the Mini International Neuropsychiatric Interview for Children and Adolescents (MINI-KID) in a clinical sample of children with mental disorders and their parents, and to examine health and demographic factors associated with agreement. METHOD: MINI-KID results were analyzed for 88 parent-child dyads. Children were between 8-17 years old and were receiving in- or outpatient services for at least one mental disorder at a pediatric hospital. Kappas were calculated to assess parent-child agreement and logistic regression models were used to identify factors associated with agreement. RESULTS: Agreement was low to moderate (κ=0.19-0.41) across the MINI-KID modules. Household income was associated with agreement for major depression, generalized anxiety, and attention-deficit hyperactivity disorder. Recruitment setting and parent psychological distress were associated with agreement for generalized anxiety and separation anxiety, respectively. Age, sex, and child disability/impairment were not associated with agreement. CONCLUSIONS: Parent-child agreement on the MINI-KID was low to moderate, and few factors were associated with agreement. These initial findings reaffirm the need for multiple informants when assessing psychopathology in children and can be used by health professionals to facilitate parent-child discussions in clinical settings in child psychiatry.

OBJECTIFS: De multiples informateurs sont souvent utilisés pour évaluer la psychopathologie de l'enfant; cependant, l'entente parent-enfant est faible en psychiatrie de l'enfant. L'objectif de la présente étude exploratoire était d'évaluer l'entente des informateurs à l'égard du mini-entretien neuropsychiatrique international pour enfants et adolescents (MINI-KID) dans un échantillon clinique d'enfants souffrant de troubles mentaux et de leurs parents, et d'examiner les facteurs de santé et démographiques associés à l'entente. MÉTHODE: Le résultats du MINI-KID ont été analysés pour 88 dyades parent-enfant. Les enfants avaient entre 8 et 17 ans et recevaient des services ambulatoires ou hospitalisés pour au moins un trouble mental dans un hôpital psychiatrique. Les kappas ont été calculés pour évaluer l'entente parent-enfant et des modèles de régression logistique ont servi à identifier les facteurs associés à l'entente. RÉSULTATS: L'entente était de faible à modérée (κ = 0,19­0,41) dans tous les modules du MINI-KID. Le revenu du ménage était associé à l'entente pour la dépression majeure, l'anxiété généralisée, et le trouble de déficit de l'attention avec hyperactivité. Les paramètres du recrutement et la détresse psychologique parentale étaient associés à l'entente pour l'anxiété généralisée et l'angoisse de séparation respectivement. L'âge, le sexe et la déficience/incapacité de l'enfant n'étaient pas associés à l'entente. CONCLUSIONS: L'entente parent-enfant au MINI-KID était faible à modérée, et peu de facteurs étaient associés à l'entente. Ces premiers résultats réaffirment le besoin de multiples informateurs pour évaluer la psychopathologie des enfants et peuvent être utilisés par les professionnels de la santé pour animer les discussions parent-enfant dans les milieux cliniques de psychiatrie de l'enfant.

Examining the Association between Community-Level Marginalization and Emergency Room Wait Time in Ontario, Canada.

OBJECTIVE: This study examines the association between community-level marginalization and emergency room (ER) wait time in Ontario. METHODS: Data sources included ER wait time data and Ontario Marginalization Index scores. Linear regression models were used to quantify the association. RESULTS: A positive association between total marginalization and overall, high-acuity and low-acuity ER wait time was found. Considering specific marginalization dimensions, we found positive associations between residential instability and ER wait time and negative associations between dependency and ER wait time. CONCLUSIONS: Reductions in community-level marginalization may impact ER wait time. Future studies using individual-level data are necessary.

Persistent ventilation inhomogeneity after an acute exacerbation in preschool children with recurrent wheezing.

BACKGROUND: Preschool children with recurrent wheezing suffer high morbidity. It is unclear whether objective measures of asthma control, such as pulmonary function tests (PFTs), provide additional information to the clinical assessment. METHODS: We recruited children between 3 and 6 years old, with a history of recurrent wheezing in the preceding year and treated for acute wheezing exacerbation in the emergency department (ED) into an observational cohort study. Children attended two outpatient visits: the first study visit within five days of discharge from the ED and the second study visit 12 weeks after the ED visit. We performed standardized symptom score (test for respiratory and asthma control in kids (TRACK)), multiple breath washout (MBW), spirometry, and clinical assessment at both visits. RESULTS: Seventy-four children, mean (standard deviation (SD)) age of 4.32 years (0.84), attended both visits. Paired FEV0.75 and lung clearance index (LCI) measurements at both time points were obtained in 37 and 34 subjects, respectively. Feasibility for all tests improved at visit 2 and was not age-dependent. At the second study visit, a third had controlled asthma based on the TRACK score, and the mean lung clearance index (LCI) improved from 9.86 to 8.31 (P = .003); however, 46% had an LCI in the abnormal range. FEV0.75 z-score improved from -1.66 to -1.17 (P = .05) but remained in the abnormal range in 24%. LCI was abnormal in more than half of the children with "well-controlled" asthma based on the TRACK score. There was no correlation between PFT measures and TRACK scores at either visit. CONCLUSIONS: Lung clearance index demonstrates a persistent deficit post-exacerbation in a large proportion of preschoolers with recurrent wheezing, highlighting that symptom scores alone may not suffice for monitoring these children.

Are Surrogate Endpoints Unbiased Metrics in Clinical Benefit Scores of the ASCO Value Framework?

BACKGROUND: Clinical benefit scores (CBS) are key elements of the ASCO Value Framework (ASCO-VF) and are weighted based on a hierarchy of efficacy endpoints: hazard ratio for death (HR OS), median overall survival (mOS), HR for disease progression (HR PFS), median progression-free survival (mPFS), and response rate (RR). When HR OS is unavailable, the other endpoints serve as "surrogates" to calculate CBS. CBS are computed from PFS or RR in 39.6% of randomized controlled trials. This study examined whether surrogate-derived CBS offer unbiased scoring compared with HR OS-derived CBS. METHODS: Using the ASCO-VF, CBS for advanced disease settings were computed for randomized controlled trials of oncology drug approvals by the FDA, European Medicines Agency, and Health Canada in January 2006 through December 2017. Mean differences of surrogate-derived CBS minus HR OS-derived CBS assessed the tendency of surrogate-derived CBS to overestimate or underestimate clinical benefit. Spearman's correlation evaluated the association between surrogate- and HR OS-derived CBS. Mean absolute error assessed the average difference between surrogate-derived CBS relative to HR OS-derived CBS. RESULTS: CBS derived from mOS, HR PFS, mPFS, and RR overestimated HR OS-derived CBS in 58%, 68%, 77%, and 55% of pairs and overall by an average of 5.62 (n=90), 6.86 (n=110), 29.81 (n=101), and 3.58 (n=108), respectively. Correlation coefficients were 0.80 (95% CI, 0.70-0.86), 0.38 (0.20-0.53), 0.20 (0.00-0.38), and 0.01 (-0.18 to 0.19) for mOS-, HR PFS-, mPFS-, and RR-derived CBS, respectively, and mean absolute errors were 11.32, 12.34, 40.40, and 18.63, respectively. CONCLUSIONS: Based on the ASCO-VF algorithm, HR PFS-, mPFS-, and RR-derived CBS are suboptimal surrogates, because they were shown to be biased and poorly correlated to HR OS-derived CBS. Despite lower weighting than OS in the ASCO-VF algorithm, PFS still overestimated CBS. Simple rescaling of surrogate endpoints may not improve their validity within the ASCO-VF given their poor correlations with HR OS-derived CBS.

Helpful ways providers can communicate about persistent medically unexplained physical symptoms.

BACKGROUND: Communication between patients and providers about persistent "medically unexplained" physical symptoms (MUS) is characterized by discordance. While the difficulties are well documented, few studies have examined effective communication. We sought to determine what veterans with Gulf War Illness (GWI) perceive as the most helpful communication from their providers. Veterans with GWI, a type of MUS, have historically had complex relationships with medical providers. Determining effective communication for patients with particularly complex relationships may help identify the most critical communication elements for all patients with MUS. METHODS: Two hundred and-ten veterans with GWI were asked, in a written questionnaire, what was the most useful thing a medical provider had told them about their GWI. Responses were coded into three categories with 10 codes. RESULTS: The most prevalent helpful communication reported by patients was when the provider offered acknowledgement and validation (N = 70). Specific recommendations for managing GWI or its symptoms (N = 48) were also commonly reported to be helpful. In contrast, about a third of the responses indicated that nothing about the communication was helpful (N = 63). There were not differences in severity of symptoms, disability or healthcare utilization between patients who found acknowledgement and validation, specific recommendations or nothing helpful. CONCLUSIONS: Previous research has documented the discord between patients and providers regarding MUS. This study suggests that most patients are able to identify something helpful a provider has said, particularly acknowledgement and validation and specific treatment recommendations. The findings also highlight missed communication opportunities with a third of patients not finding anything helpful.

Do older and younger patients derive similar survival benefits from novel oncology drugs? A systematic review and meta-analysis.

Background: older patients are commonly believed to derive less benefit from cancer drugs, even if they fulfil clinical trial eligibility [Talarico et al. (2004, J Clin Oncol, 22(22):4626-31)]. We aim to examine if novel oncology drugs provide differential age-based treatment outcomes for patients on clinical trials. Methods: a systematic review of randomised control trials (RCTs) cited for clinical efficacy evidence in novel oncology drug approvals by the Food and Drug Administration, European Medicines Agency and Health Canada between 2006 and 2017 was conducted. Studies reporting age-based subgroup analyses for overall or progression-free survival (OS/PFS) were included. Hazard ratios (HRs) and confidence intervals (CIs) for age-based subgroups were extracted. Meta-analyses with random effects were conducted, examining patient subgroups <65 and ≥65 years separately and pooled HRs of studies primary endpoints (OS or PFS) compared to examine if differences existed between age-based subgroups. Sensitivity analyses were conducted for cancer type, primary endpoint and systemic treatment. Results: one-hundred-two RCTs, including 65,122 patients, met the inclusion criteria. One study reported age-based toxicity and none reported age-based quality of life (QOL) results. Pooled HRs [95% CIs] for patients <65 and ≥65 years were 0.61 [0.57-0.65] and 0.65 [0.61-0.70], respectively, with no difference between them (P = 0.14). Sensitivity analyses revealed similar results. Conclusion: our results suggest that older and young patients, who fulfil clinical trial eligibility, may derive similar relative survival benefits from novel oncology drugs. There is, however, a need to report age-based toxicity and QOL results to support patient discussions regarding the balance of treatment benefit and harm, to encourage informed decision-making.

Examining Trends in Cost and Clinical Benefit of Novel Anticancer Drugs Over Time.

PURPOSE: The purpose of this study was to determine if clinical benefits of novel anticancer drugs, measured by the ASCO Value Framework and European Society of Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale, have increased over time in parallel with increasing costs. METHODS: Anticancer drugs from phase III randomized controlled trials cited for clinical efficacy evidence in drug approvals between January 2006 to December 2015 were identified and scored using both frameworks. For each drug, the monthly price and incremental anticancer drug costs were calculated. Relationships between cost and year of approval were examined using generalized linear regressions models. Ordinary least square models were used to evaluate relationships between ASCO and ESMO scores and year of approval. Spearman correlation coefficients between costs and clinical benefit scores were calculated. RESULTS: In total, 42 randomized controlled trials were included. Both monthly prices and incremental anticancer drug costs were significantly associated with year of approval and showed an average annual increase of 9% and 21%, respectively. The predicted mean incremental anticancer drug cost increased from $30,447 in 2006 to $161,141 in 2015 (greater than five-fold increase). Both ASCO and ESMO scores were not statistically associated with year of approval or correlated with monthly prices or incremental anticancer drug costs. CONCLUSION: Over the past decade, costs of novel oncology drugs have increased, while clinical benefits of these medications have not experienced a proportional positive change. The incremental anticancer drug costs have increased at a much greater rate than monthly prices, indicating that the increase in anticancer drug costs may be higher than commonly reported.

Do the American Society of Clinical Oncology Value Framework and the European Society of Medical Oncology Magnitude of Clinical Benefit Scale Measure the Same Construct of Clinical Benefit?

Purpose Whether the ASCO Value Framework and the European Society for Medical Oncology (ESMO) Magnitude of Clinical Benefit Scale (MCBS) measure similar constructs of clinical benefit is unclear. It is also unclear how they relate to quality-adjusted life-years (QALYs) and funding recommendations in the United Kingdom and Canada. Methods Randomized clinical trials of oncology drug approvals by the US Food and Drug Administration, European Medicines Agency, and Health Canada between 2006 and August 2015 were identified and scored using the ASCO version 1 (v1) framework, ASCO version 2 (v2) framework, and ESMO-MCBS by at least two independent reviewers. Spearman correlation coefficients were calculated to assess construct (between frameworks) and criterion validity (against QALYs from the National Institute for Health and Care Excellence [NICE] and the pan-Canadian Oncology Drug Review [pCODR]). Associations between scores and NICE/pCODR recommendations were examined. Inter-rater reliability was assessed using intraclass correlation coefficients. Results From 109 included randomized clinical trials, 108 ASCOv1, 111 ASCOv2, and 83 ESMO scores were determined. Correlation coefficients for ASCOv1 versus ESMO, ASCOv2 versus ESMO, and ASCOv1 versus ASCOv2 were 0.36 (95% CI, 0.15 to 0.54), 0.17 (95% CI, -0.06 to 0.37), and 0.50 (95% CI, 0.35 to 0.63), respectively. Compared with NICE QALYs, correlation coefficients were 0.45 (ASCOv1), 0.53 (ASCOv2), and 0.46 (ESMO); with pCODR QALYs, coefficients were 0.19 (ASCOv1), 0.20 (ASCOv2), and 0.36 (ESMO). None of the frameworks were significantly associated with NICE/pCODR recommendations. Inter-rater reliability was good for all frameworks. Conclusion The weak-to-moderate correlations of the ASCO frameworks with the ESMO-MCBS, as well as their correlations with QALYs and with NICE/pCODR funding recommendations, suggest different constructs of clinical benefit measured. Construct convergent validity with the ESMO-MCBS did not increase with the updated ASCO framework.